Orphan Designation of ATMPs for Rare Diseases: MPS II Case Study Many advanced therapy medicinal products (ATMPs) in development in the EU are for rare diseases and conditions. Since the...
Pharmaceutical companies should understand EU Health Technology Assessment (HTA) authorities requirements early in the product development phase. Engagement with HTA authorities during clinical...
Regulatory Sciences
If a marketing authorisation is planned to be submitted in England, Scotland, and Wales (GB), an MHRA-approved paediatric investigational plan (PIP) is required. Up until January 1, 2021, PIPs were...
What is ILAP? What benefits does ILAP provide? How do you access it? With the dust of Brexit settling, the question on most people’s lips (well, those of us in the healthcare sector anyway!) was:...
For the first time, the FDA has issued a draft guidance for industry on “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics”. Oligonucleotides are short single...
A unique opportunity to interact with medicine regulators in Europe Are you considering requesting scientific advice in Europe? We can help you navigate the various procedures within the European...
From FDA's approval of the first cannabis-based product in the U.S., to the classification of two Apple Watch apps and changes in the Agency's submission requirements related to Sponsor meetings,...
The Prescription Drug User Fee Act (PDUFA) is due to be reauthorized by the end of September this year. Along with the usual increases in fees, the latest version of the act (PDUFA V) includes some...